Systematic review of ampullary neuroendocrine tumors: a pooled analysis of 32 cases reported from 2010 to 2024

Ampullary neuroendocrine tumors (NETs) are rare and heterogeneous neoplasms, and their diagnosis and management remain a significant challenge. This study aims to systematically analyze the cases reported in the literature over the 14-year period from 2010 to 2024, and summarize the clinical characteristics, diagnostic and therapeutic strategies, as well as prognostic status of neuroendocrine tumors of the ampulla of Vater. A systematic literature search was conducted for studies on ampullary NETs published between January 2010 and June 2024. Clinical and pathological data, imaging findings, treatment regimens, and follow-up outcomes of patients were collected and retrospectively analyzed. A total of 16 studies involving 32 patients were included. There were 19 male and 13 female patients, with a male-to-female ratio of 1.46:1. The median age at diagnosis was 56 years (range: 18–77 years). The most common clinical presentations were abdominal pain (35.5%) and jaundice (22.5%), while 25.8% of cases were incidentally detected during routine health examinations. Computed tomography (CT) (90.6%) and magnetic resonance imaging (MRI) (37.5%) were the primary imaging modalities used. Endoscopic ultrasound (EUS)- and endoscopic retrograde cholangiopancreatography (ERCP)-guided biopsies both demonstrated a diagnostic accuracy of 100%. The median tumor diameter was 15.5 mm. Both larger tumor size and greater depth of invasion were associated with a higher rate of lymph node metastasis. Treatment strategies included endoscopic resection (31.3%) and surgical resection (59.4%). The median follow-up duration was 38.9 months, with a 5-year overall survival rate of 96.4%. Ampullary NETs present with nonspecific clinical manifestations and are prone to being overlooked or misdiagnosed. EUS- or ERCP-guided biopsy can significantly improve preoperative diagnostic accuracy. The study findings indicate that tumor size, depth of invasion, and tumor grade are associated with the risk of lymph node metastasis. Treatment decisions should be individualized based on the aforementioned characteristics and in accordance with the principle of multidisciplinary collaboration: endoscopic resection is suitable for carefully selected early-stage cases, whereas radical surgery remains the core therapeutic modality for high-risk or advanced-stage diseases. Although the overall prognosis is favorable, the risk of recurrence persists; therefore, establishing a long-term regular follow-up system is crucial, especially for patients with high-risk pathological features. Given that the conclusions of this study are based on small-sample retrospective data, the relevant correlations need to be further verified by larger-scale prospective studies. Not applicable.